EMEA-002830-PIP01-20 - paediatric investigation plan

A self-complementary adeno-associated virus [AAV] serotype 8 virus particle encoding the human ornithine transcarbamylase [OTC] gene sequence (DTX301)

Key facts

• Active substance:
  A self-complementary adeno-associated virus [AAV] serotype 8 virus particle encoding the human ornithine transcarbamylase [OTC] gene sequence (DTX301)
• Therapeutic area:
  Endocrinology-Gynaecology-Fertility-Metabolism
• Decision number:
  P/0432/2023
• PIP number:
  EMEA-002830-PIP01-20
• Pharmaceutical form(s):
  Concentrate for solution for infusion
• Condition(s) / indication(s):
  Treatment of ornithine transcarbamylase deficiency
• Route(s) of administration:
  Intravenous use
• Decision type:
  P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)
• Compliance check done:
  No

Decision

P/0432/2023 : EMA decision of 27 October 2023 on the agreement of a paediatric investigation plan and on the granting of a deferral and on the granting of a waiver for self-complementary adeno-associated virus [AAV]......(DTX301) (EMEA-002830- PIP01-20)

Adopted Reference Number: EMA/470006/2023